Acid Sphingomyelinase Deficiency
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Clinical trial update for GammaGA examining how common acid sphingomyelinase deficiency (ASMD) and Gaucher disease are in patients with mono…
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CLINICAL_TRIALS research · 2025-12-10Clinical Trial Update: Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
The entry reports an update to the diagnostic criteria for Acid Sphingomyelinase Deficiency (ASMD) in an active, non-recruiting clinical tri…
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CLINICAL_TRIALS research · 2025-10-31Clinical Trial Update: A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
This is an ongoing real-world, long-term safety and immunogenicity study of olipudase alfa in infants under 2 years with acid sphingomyelina…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
A non-interventional national pediatric study sponsored by Sanofi investigating unexplained enlarged spleen in patients with Gaucher disease…
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CLINICAL_TRIALS research · 2025-05-22Clinical Trial Update: National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
A national multicentre observational study investigating the natural history of acid sphingomyelinase deficiency (ASMD) in adults and childr…
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